Ultragenyx Shares Fall After Phase 3 Aspire Study Misses Primary Endpoint

Ultragenyx (NASDAQ:RARE) shares fell 44.7% to $14.67 in premarket trading after the company disclosed that its pivotal Phase 3 Aspire study evaluating apazunersen (GTX-102) in Angelman syndrome did not meet its primary or key secondary endpoints.

The company announced the results late Wednesday. The study’s primary endpoint measured the change from baseline in the Bayley-4 cognitive raw score, while the key secondary endpoint assessed net response using the Multidomain Responder Index.

According to the source material, investigators found no meaningful differences between the treatment and placebo groups on either measure.

Analysts Cut Ratings and Price Targets

Several analysts revised their ratings and price targets for Ultragenyx following the study results.

Evercore ISI downgraded Ultragenyx from Outperform to In Line and reduced its price target to $16 from $34.

Baird lowered its rating from Outperform to Neutral and cut its price target to $16 from $40.

JPMorgan downgraded the stock from Overweight to Neutral and reduced its price target to $36 from $80.

William Blair also downgraded Ultragenyx shares to Market Perform. According to the source material, the firm removed the contribution from GTX-102 from its pipeline valuation and cited the company’s ongoing cash consumption and the longer-term risk associated with Crysvita losing exclusivity.

Shares Trade Below Previous 52-Week Low

The broader US equity market was fractionally higher, with the S&P 500, Dow Jones and Nasdaq recording modest gains.

According to the source material, there were no major central bank announcements or economic data releases materially affecting biotechnology-sector sentiment during the session.

Following the premarket decline, Ultragenyx shares were trading below their previous 52-week low of $18.29 and at their lowest level in more than a year.

Ultragenyx stock price